Carthera, a clinical-stage medtech company spun off from Sorbonne University, has received European Medicines Agency (EMA) expert panel endorsement for Orphan Medical Device status of its SonoCloud ultrasound system, positioning the company as one of the first to benefit from the EU's new pilot program supporting innovative devices for rare diseases.
The endorsement provides Carthera with access to expert panel advice on clinical development strategy and clarifies the final steps before CE marking application. This guidance is part of the EU pilot program launched in August 2024 to support early access pathways for innovative devices targeting rare diseases, with the EMA prioritizing devices treating life-threatening conditions or those that could cause permanent impairment of body function.
Revolutionary Blood-Brain Barrier Technology
SonoCloud is an intracranial implant that emits ultrasound to temporarily increase the permeability of blood vessels in the brain, addressing a critical challenge in neurological treatment. Past research has demonstrated that the blood-brain barrier, the brain's natural filtration system, limits the penetration of more than 98% of drugs.
The device is inserted into the skull and activated prior to injection of therapeutic agents. Several minutes of low-intensity ultrasound opens the blood-brain barrier for six hours, significantly increasing the concentration of therapeutic molecules in the brain. This ultrasound-induced opening of the blood-brain barrier represents a world first in medical technology.
Promising Clinical Results in Glioblastoma
Carthera is currently evaluating SonoCloud for improving treatment of recurrent glioblastoma (rGBM), one of the most aggressive brain cancers. In 2024, the company reported results from a Phase I/II single-arm trial (NCT03744026) assessing the safety and efficacy of blood-brain barrier disruption with the implantable ultrasound system in rGBM patients receiving carboplatin.
The trial demonstrated encouraging survival outcomes in 12 patients who received carboplatin just before using the device. The one-year overall survival rate was 58%, with a median overall survival of 14 months from surgery. These results compare favorably to the historical survival rate for patients with recurrent glioblastoma, which ranges from 9-11 months.
Regulatory Pathway and Future Development
"This is a very important step for the company, since it creates a unique forum for dialogue with the expert panel prior to submitting the technical documentation for conformity assessment," said Sandra Thiollière, director of regulatory affairs at Carthera. "In parallel, we are continuing our early-stage contacts with our notified body in order to prepare for CE marking submission."
The next step for Carthera involves benefiting from free advice from the expert panel on data needed to complete clinical evaluation of the SonoCloud device, scheduled for Q3 2025. The company is also continuing structured dialogue with its notified body to prepare technical documentation for CE marking submission.
Pivotal Trial and Market Positioning
Carthera is currently recruiting patients for a pivotal trial of SonoCloud. The two-arm study will enroll 560 patients and evaluate the system combined with carboplatin versus standard of care treatments, including lomustine or temozolomide, in patients undergoing planned resection for first recurrence glioblastoma.
"In 2022, we obtained Breakthrough Device Designation from the FDA, and in 2024, the Forfait Innovation in France from the HAS. With this new status, we will be in the best position to put our highly promising technique on the market as early as possible and to bring its benefits to European patients," said Frédéric Sottilini, CEO of Carthera.
The company has previously received FDA Breakthrough Device Designation in 2022 and FDA/EMA Orphan Drug Designation in 2023 for carboplatin when used with SonoCloud, establishing a strong regulatory foundation across major markets.
Technology Applications and Company Background
Founded in 2010 by Professor Alexandre Carpentier, a head neurosurgeon at AP-HP Sorbonne University, Carthera leverages inventions that have achieved worldwide recognition for innovative developments in treating brain disorders. The SonoCloud technology was developed in collaboration with the Laboratory of Therapeutic Applications of Ultrasound (LabTAU, INSERM) in Lyon, France.
The ultrasound-based approach offers potential treatment options for a wide range of indications beyond brain tumors, including neurodegenerative diseases. Since its inception, the technical and clinical development of SonoCloud has received support from multiple organizations including the National Research Agency (ANR), the French public investment bank (Bpifrance), the National Institutes of Health (NIH), and the European Innovation Council (EIC).
